Drugs
FDA has lifted a 2022-ordered partial clinical hold on PTC Therapeutics’ PTC518 for treating Huntington’s disease based on the agency’s review of Phase 2 trial data (PIVOT-HD). The company says interim results from the study showed that at “month 12, PTC518 treatment resulted in dose-dependent
Drugs
FDA has approved a major modification to the Risk Evaluation and Mitigation Strategy (REMS) for Kite Pharma’s lymphoma drugs Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel). An agency notice says FDA has determined that the REMS must be modified to minimize the burden on the healthcare delivery system of
Drugs
A 5/30-6/7 FDA inspection at the Dr. Reddy’s Laboratories active pharmaceutical ingredient manufacturing facility in Andhra, Pradesh, India has four inspection observations. FDA has released the form FDA-483 issued following the inspection with these observations: * failure to validate or verify analytical methods under the actual
Drugs
FDA has published a guidance, Circumstances that Constitute Delaying, Denying, Limiting, or Refusing a Drug or Device Inspection, meeting a requirement for such a guidance in the 2012 FDA Safety and Innovation Act. The document finalizes a 12/16/2022 draft guidance and supersedes a 10/2014 guidance, which is
Drugs
Jazz Pharmaceuticals says top-line results from its Phase 2b clinical trial (NCT05122650) evaluating the efficacy and safety of suvecaltamide in adult patients with essential tremor did not achieve statistical significance at 30mg versus placebo on the primary endpoint of change from baseline to week 12 on the Essential Tremor
Drugs
Johnson & Johnson has submitted an sBLA to FDA for approval of its Tremfya (guselkumab) to treat adults with moderately to severely active Crohn’s disease. A company statement says that it also submitted an sBLA in March for an indication to treat severely active ulcerative colitis. The Crohn’s
Drugs
FDA has approved a new indication for AbbVie’s Skyrizi (risankizumab-rzaa) to treat moderate to severe ulcerative colitis. The company says the approval was supported by two Phase 3 trials, the INSPIRE 12-week induction trial and the COMMAND 52-week maintenance study. Skyrizi is described as an interleukin-
Drugs
FDA is notifying NDA and ANDA sponsors that clinical and bioanalytical studies conducted by Synapse Labs Pvt. Ltd. in Pune, India, are not acceptable due to data integrity concerns. An agency online notice says that studies conducted by Synapse must be repeated. “During analyses of study data generated at Synapse
Drugs
Several stakeholders are expressing their appreciation for FDA’s issuance of a draft guidance, Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products. Flatiron Health says the draft will give sponsors and real-world data (RWD) organizations clarity in regulatory expectations in designing a non-interventional
Drugs
FDA has published a report on the topics discussed at an 11/29-30/2023 public workshop it held with the Clinical Trials Transformation Initiative to solicit input on increasing the enrollment of historically underrepresented populations in clinical studies and encouraging clinical study participation that reflects the prevalence of the
Drugs
Researchers at Rutgers University and the University of California San Diego say that open access to three-dimensional atomic-level biostructure information from the Protein Data Bank (PDB) facilitated the discovery/development of 100% of the 34 new low molecular weight, protein-targeted, antineoplastic agents approved by FDA between 2019
Drugs
AstraZeneca says its CAPItello-290 Phase 3 trial of Truqap (capivasertib) in combination with paclitaxel in patients with locally advanced or metastatic triple-negative breast cancer did not meet dual primary endpoints of improvement in overall survival versus paclitaxel in combination with placebo in either the overall trial population or
Drugs
FDA has placed a partial clinical hold on Zentalis Pharmaceuticals’ azenosertib studies, including a Phase 1 (ZN-c3-001) dose-escalation study in solid tumors, a Phase 2 (DENALI) study in platinum-resistant ovarian cancer, and a Phase 2 (TETON) study in uterine serous carcinoma. The action is in response
Drugs
Takeda says topline data from its SKYLINE and SKYWAY studies of soticlestat (TAK-935) failed to meet their primary endpoints. The Phase 3 SKYLINE (TAK-935-3001) study evaluated soticlestat plus standard of care versus placebo plus standard of care in patients with refractory Dravet syndrome. “Soticlestat narrowly missed the
Drugs
FDA has placed a partial clinical hold on a BioNTech/Medilink Therapeutics Phase 1 clinical (NCT05653752) trial that is evaluating an early-stage antibody-drug conjugate (ADC) product candidate (BNT326/YL202) as a later-line treatment in heavily pre-treated patients with advanced or metastatic epidermal growth factor receptor-mutated
Drugs
FDA has approved Merck’s Keytruda (pembrolizumab) for use with carboplatin and paclitaxel, followed by single-agent pembrolizumab in adult patients with primary advanced or recurrent endometrial carcinoma. Approval was based on data from the KEYNOTE-868/NRG-GY018 (NCT03914612) trial that enrolled 810 patients with advanced or recurrent endometrial
Drugs
FDA has released a final guidance entitled “Facility Readiness: Goal Date Decisions Under GDUFA” that provides information on how the agency intends to assign a generic drug ANDA goal date based on a facility’s readiness for inspection. The document explains how FDA deals with a “program enhancement” agreed upon
Drugs
FDA has issued a one-item Form FDA-483 to Chugai Pharma Manufacturing Co. after inspecting the firm’s Utsunomiya, Tochigi, Japan facility 3/25 to 4/2. The agency cited Chugai over its procedures for preventing microbiological contamination of drug products because they did not include adequate validation of
Drugs
FDA has released a final guidance (XXINSERT LINKXX) entitled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics.” The agency says oligonucleotide therapeutics are “an emerging therapeutic modality with increasing numbers of drugs in development.” The document provides recommendations for certain evaluations during development of oligonucleotide therapeutics, including: (1) characterizing
Drugs
FDA has posted a final guidance entitled “Diabetic Foot Infections (DFI): Developing Drugs for Treatment.” The document addresses the agency’s current thinking regarding the overall development program and clinical trial designs for developing drugs to support an indication for treating DFI. FDA says that important considerations for developing such
Drugs
FDA has approved an expanded use for Amgen’s Blincyto (blinatumomab) for treating adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia in the consolidation phase, regardless of measurable residual disease status. The company says the approval is based
Drugs
CDER says it has added three examples of Bayesian statistical analysis plans to its Center for Clinical Trial Innovation Supplemental Analysis Demonstration Project Web page. An online post says the demonstration project “seeks to advance the understanding of Bayesian statistical methods and establish processes for integrating these approaches into clinical